The present invention generally relates to treating muscular wasting diseases by inhibiting the NF-κB pathway.
Muscular wasting diseases, such as muscular dystrophies, are a group of degenerative diseases that culminate in progressive skeletal muscle wasting leading to muscle weakness, wheelchair dependence, and in some cases death. Of the muscular dystrophies, Duchenne muscular dystrophy is the most severe and most widely recognized. Duchenne muscular dystrophy occurs in one in 3500 male births per year and is the most frequent of all lethal X-linked recessive diseases. Duchenne muscular dystrophy symptoms typically appear in boys by the ages of 3 to 5 years old. By their teenage years these affected boys are wheelchair bound, and by their second or third decade of life, they usually succumb to the disease due to diaphragm and/or cardiac muscle dysfunction.
Another muscular wasting disease, which shows similar symptoms, although less severe, as Duchenne muscular dystrophy, is Becker muscular dystrophy. Even though the defective dystrophin gene causing both Duchenne muscular dystrophy and Becker muscular dystrophy has been known for over 20 years, a cure is still lacking.
Also included in the class of muscular dystrophies, yet not as well-known as Duchenne muscular dystrophy or Becker muscular dystrophy, is Limb Girdle muscular dystrophy. Specifically, for many types of Limb Girdle muscular dystrophy, the defective gene causing the disease is not yet known.